>> NEUROBLASTOMA IS A CANCER OF CHILDHOOD.
IT'S THE MOST COMMON CANCER WE SEE IN CHILDREN WHO
ARE UNDER THE AGE OF ONE.
AND OTHER THAN BRAIN TUMORS, IT'S THE MOST COMMON SOLID
CANCER THAT WE SEE IN CHILDREN.
THERE HAVE BEEN SOME RECENT PROGRESS AS WE'VE
INTENSIFIED THE TREATMENT AND ALSO BECOME A BIT
SMARTER ABOUT HOW WE GIVE THAT TREATMENT.
BUT STILL TODAY IN THE YEAR 2012,
HALF OF CHILDREN WITH THE AGGRESSIVE FORM OF THIS
DISEASE SUFFER A RELAPSE.
THAT IS WHAT FUELS MY RESEARCH.
IN OUR LAB WE ARE DEDICATED TO MAKING DISCOVERIES
THAT WILL IMPACT THE CLINIC.
AND WE HAVE BEEN FOCUSED ON THE RARE FAMILIES WHERE
NEUROBLASTOMA IS INHERITED, KNOWING FROM OTHER DISEASES
LIKE BREAST CANCER THAT THESE FAMILIES CAN TEACH US
ABOUT WHAT DRIVES THE CANCER.
AND IN 2008, WE MADE THE DISCOVERY THAT THESE
FAMILIES ALL HAVE ABNORMALITIES IN THE DNA
OF THIS GENE WE NOW CALL ALK, WHICH STANDS FOR
ANAPLASTIC LYMPHOMA KINASE.
IT'S A GENE THAT WHEN IT'S TURNED ON,
IT ALLOWS THE TUMOR TO GROW.
AND SO FORTUNATE FOR US, IT ALLOWS US THE OPPORTUNITY
TO THINK OF WAYS TO TURN IT OFF.
>> THAT WAS FOUND TO BE THE CAUSE OF WHY CERTAIN
CHILDREN DEVELOPED NEUROBLASTOMA.
BUT, ALSO, WAS FOUND TO BE MUTATED IN THE ACTUAL TUMOR
CANCER CELLS AND, THEREFORE, PERHAPS WAS AN ACHILLES' HEEL,
SOMETHING THAT WAS MAKING THE CANCERS GROW
ABNORMALLY AND, THEREFORE, CAN BE TARGETED
WITH THE NEW TREATMENT.
IF IT IS A MUTATION THAT IS MAKING THE CANCER CELL WANT
TO GROW AND DIVIDE AND KEEP ON SPREADING,
THEN ONE CAN FIGURE OUT A WAY TO TAKE THAT "ON" SWITCH
FOR A CANCER CELL AND TURN IT OFF.
>> OF THE EIGHT PATIENTS WITH LYMPHOMA WHO WENT ON,
SEVEN OF THE EIGHT HAVE HAD SUBSTANTIAL BENEFIT
FROM THIS DRUG AND THEIR SYMPTOMS CLEARED WITHIN DAYS
OR A COUPLE OF WEEKS OF STARTING THIS PILL.
AND NOW ALL OF THESE PATIENTS REMAIN WITHOUT ANY EVIDENCE
OF ACTIVE DISEASE.
FOR THE IMT PATIENTS, I CAN SAY THAT THEY'VE ALL
BENEFITED IN ONE FORM OR THE OTHER.
THEY'VE HAD EITHER TUMOR SHRINKAGE OR SOME OBJECTIVE
RESPONSE AND ALL OF THESE HAVE BEEN PATIENTS REMAIN
ON TREATMENT WITH THE DRUG.
AND THEN FOR NEUROBLASTOMA, THE EIGHT WHO HAD AN ALK
MUTATION THAT WE KNOW OF, FIVE OF THOSE DID NOT RESPOND.
ALL FIVE OF THOSE PATIENTS WERE TREATED AT LOWER DOSES.
OF THE THREE OTHERS WITH A KNOWN ALK MUTATION,
ONE OF THEM REMAINS ON THE DRUG AFTER BEING ON IT
FOR 10 MONTHS AND HAS DISEASE THAT HAS BEEN QUIET
AND THE PATIENT HAS EXCELLENT QUALITY OF LIFE,
AND THE OTHER TWO ARE PATIENTS WHO HAVE HAD
COMPLETE RESPONSES.
WE THINK THIS IS VERY PROMISING FOR KIDS WHO
HISTORICALLY HAVE HAD VERY FEW OPTIONS AND LITTLE HOPE.
THIS IS A FIRST.
IT'S A FIRST FOR CHILDREN WHO HAVE THIS RARE FORM
OF CHILDHOOD CANCER TO BE ABLE TO TAKE A PILL,
TWICE A DAY, AND TURN OFF THEIR CANCER.
>> THE INTEGRATION OF THIS PRECISION TYPE OF MEDICINE
INTO FRONTLINE THERAPY IS A SHINING EXAMPLE OF THE POWER
OF MODERN CANCER RESEARCH.
>> WE'RE NOW MOVING INTO AN ERA WHERE THE GOAL IS TO
MAKE A BIG DIFFERENCE FOR A SMALL GROUP OF PATIENTS
RATHER THAN MAKING A SMALL DIFFERENCE FOR A BIG GROUP
OF PATIENTS.
IN THE PAST, WE MADE A SMALL DIFFERENCE BY GIVING DRUGS
THAT ARE POISON FOR ALL THE CELLS IN OUR BODY AND AREN'T
SELECTIVE FOR THE CELLS THAT ARE REALLY THE ONES
RESPONSIBLE FOR CAUSING THE CANCER.
NOW, WE'RE MOVING INTO AN ERA WHERE THE GOAL
IS TO SPECIFICALLY, FIRST AND FOREMOST,
DISCOVER WHAT ARE THOSE CELLS FUELING THE CANCER.
AND FOR A SUBSET OF PATIENTS IT'S ALK.
AND WE CAN NOW PROVIDE VERY SPECIFIC TAILORED THERAPY,
INDEED PERSONALIZED MEDICINE, TO IMPROVE
OUR CHANCE OF CURING THESE PATIENTS.